Aplastic Anemia & MDS International Foundation 2012 Bone Marrow Failure Disease Scientific Symposium Project Abstract Aplastic anemia (AA), myelodysplastic syndromes (MDS), and paroxysmal nocturnal hemoglobinuria (PNH) are rare diseases that result in bone marrow failure. When the marrow fails to produce blood cells, the resulting anemia, thrombocytopenia (shortage of platelets in the bloodstream), and neutropenia (neutrophil shortage) can lead to debilitating symptoms and death. Once considered distinct, these three diseases are now believed to be linked by similar pathophysiologies. Exploration of current research issues in AA, MDS, and PNH would greatly benefit from increased collaboration between basic and clinical scientists and scientists studying the individual diseases. Increased understanding of the molecular events driving these diseases and of the response to treatment are needed to define at-risk populations and improve current therapies. The Aplastic Anemia & MDS International Foundation, Inc. (AA&MDSIF) proposes to hold its third Bone Marrow Failure Disease Scientific Symposium in March 2012, with Richard M. Stone, M.D., from the Dana-Farber Cancer Institute, as Principal Investigator and Co-Chair of the Symposium and Neal S. Young, M.D., from the National Heart, Lung, and Blood Institute, as Co-Chair. The Symposium will bring together physicians treating these diseases and laboratory researchers studying the immunology and cell biology of bone marrow failure to discuss current areas of controversy, share recent research results, and propose recommendations for the highest priority directions for basic and clinical research needed to advance the field. The format will consist of six presentation sessions, with time at the end of each session for questions and discussions, as well as for capture of recommendations for future research. The two days of the conference will also include poster and discussion sessions and networking opportunities. AA&MDSIF will publish lay versions of the proceedings, including research recommendations, on its website and meeting leaders will publish a summary of the proceedings for researchers and clinicians in a peer-reviewed journal. PUBLIC HEALTH RELEVANCE: Aplastic Anemia & MDS International Foundation 2012 Bone Marrow Failure Disease Scientific Symposium Project Narrative Although rare, aplastic anemia, myelodysplastic syndromes, and paroxysmal nocturnal hemoglobinuria cause significant morbidity and mortality. Collaborative efforts between physicians treating these diseases and laboratory scientists studying the pathophysiology of bone-marrow failure are needed to speed the development of effective new therapies. The Aplastic Anemia & MDS International Foundation, Inc. proposes to hold its third Bone Marrow Failure Disease Scientific Symposium in March 2012 to bring together physicians and laboratory researchers to discuss current controversies, share recent research results, and propose recommendations to advance the field.